European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibro...
August 21 2020 - 9:07AM
Business Wire
– For the first time, up to 10,000 people in
Europe ages 12 years and older with one F508del mutation and one
minimal function mutation will be eligible for a medicine that
treats the underlying cause of cystic fibrosis –
– People 12 years of age and older who have two
F508del mutations will also be eligible for the new triple
combination regimen –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission (EC) has granted marketing
authorization for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a
combination regimen with ivacaftor to treat people with cystic
fibrosis (CF) ages 12 years and older with one F508del mutation and
one minimal function mutation (F/MF), or two F508del mutations
(F/F) in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene.
For the first time, up to 10,000 people in Europe ages 12 years
and older with CF who have one F508del mutation and one minimal
function mutation will be eligible for a CFTR modulator that treats
the underlying cause of the disease. Approval of the triple
combination regimen also expands the number of treatment options
available to people ages 12 years and older with CF who have two
copies of the F508del mutation, the most common CF-causing mutation
worldwide.
“Today is a significant day for those with CF, their families
and Vertex, and one that brings us one step closer towards our
ultimate goal of discovering and developing treatments for all
patients with CF,” said Reshma Kewalramani, M.D., Chief Executive
Officer and President, Vertex. “I would like to thank our dedicated
scientists, as well as study investigators and people with CF who
participated in our clinical trials to enable this innovative
medicine to be approved in Europe today. Without their commitment,
this milestone would not have been possible.”
As a result of long-term reimbursement agreements in England,
Denmark and the Republic of Ireland, and provisions for access in
health care systems such as Germany, eligible patients in these
countries will have access to the triple combination regimen in the
upcoming weeks. Vertex is committed to working closely with
national health authorities and governments in all other countries
in Europe to secure access for eligible patients as quickly as
possible.
Marketing authorization was based on the results of two global
Phase 3 studies, which showed statistically significant and
clinically meaningful improvements in lung function (primary
endpoint) and all key secondary endpoints, in people with CF ages
12 years and older with one F508del mutation and one minimal
function mutation or two F508del mutations in the CFTR gene. The
triple combination regimen was generally well tolerated in both
studies.
“The triple combination regimen has been shown to have a major
impact on several outcome measures in people with CF,” said
Professor Harry Heijerman, Professor and Head of the Department of
Pulmonology at University Medical Center Utrecht, Netherlands. “The
clinical data showed significant improvements in lung function and
other important measures, such as sweat chloride levels and quality
of life as measured by the CFQ-R respiratory domain score, in
patients treated with the triple combination therapy. I now look
forward to seeing the impact of the medicine in clinical
practice.”
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease
affecting approximately 75,000 people worldwide. CF is a
progressive, multi-system disease that affects the lungs, liver, GI
tract, sinuses, sweat glands, pancreas and reproductive tract. CF
is caused by a defective and/or missing CFTR protein resulting from
certain mutations in the CFTR gene. Children must inherit two
defective CFTR genes — one from each parent — to have CF. While
there are many different types of CFTR mutations that can cause the
disease, the vast majority of all people with CF have at least one
F508del mutation. These mutations, which can be determined by a
genetic test, or genotyping test, lead to CF by creating
non-working and/or too few CFTR proteins at the cell surface. The
defective function and/or absence of CFTR protein results in poor
flow of salt and water into and out of the cells in a number of
organs. In the lungs, this leads to the buildup of abnormally
thick, sticky mucus that can cause chronic lung infections and
progressive lung damage in many patients that eventually leads to
death. The median age of death is in the early 30s.
About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a
Combination Regimen With ivacaftor
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination
regimen with ivacaftor 150 mg was developed for the treatment of
cystic fibrosis (CF) in patients ages 12 years and older with one
F508del mutation and one minimal function mutation (F/MF) or two
F508del mutations (F/F) in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene. KAFTRIO® is designed to increase
the quantity and function of the F508del-CFTR protein at the cell
surface. The EU submission for KAFTRIO® was supported by positive
results of two global Phase 3 studies in people ages 12 years and
older with CF: a 24-week Phase 3 study in 403 people with one
F508del mutation and one minimal function mutation (F/MF) and a
four-week Phase 3 study in 107 people with two F508del mutations
(F/F).
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious diseases. The company has multiple approved medicines
that treat the underlying cause of cystic fibrosis (CF) — a rare,
life-threatening genetic disease — and has several ongoing clinical
and research programs in CF. Beyond CF, Vertex has a robust
pipeline of investigational small molecule medicines in other
serious diseases where it has deep insight into causal human
biology, including pain, alpha-1 antitrypsin deficiency and
APOL1-mediated kidney diseases. In addition, Vertex has a rapidly
expanding pipeline of genetic and cell therapies for diseases such
as sickle cell disease, beta thalassemia, Duchenne muscular
dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global
headquarters is now located in Boston's Innovation District and its
international headquarters is in London, UK. Additionally, the
company has research and development sites and commercial offices
in North America, Europe, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including 10 consecutive years on Science magazine's Top
Employers list and top five on the 2019 Best Employers for
Diversity list by Forbes.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995,
including, without limitation, statements made by Dr. Reshma
Kewalramani and Professor Harry Heijerman in this press release,
statements regarding the eligible patient population in Europe, our
expectations regarding the timing of access to the triple
combination regimen across countries in Europe, and our plans to
secure access to our medicine for additional patients in Europe.
While Vertex believes the forward-looking statements contained in
this press release are accurate, these forward-looking statements
represent the company's beliefs only as of the date of this press
release and there are a number of factors that could cause actual
events or results to differ materially from those indicated by such
forward-looking statements. Those risks and uncertainties include,
among other things, that data from the company's development
programs may not support registration or further development of its
compounds due to safety, efficacy or other reasons, risks related
to commercializing medicines in Europe, and other risks listed
under Risk Factors in Vertex's annual report and subsequent
quarterly reports filed with the Securities and Exchange Commission
and available through the company's website at www.vrtx.com. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
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